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Oral Treprostinil in Subjects With Pulmonary Hypertension Associated With Heart Failure With Preserved Ejection Fraction
Phase 3 trial testing Oral treprostinil in Pulmonary Hypertension in 84 participants. Terminated before completion.
| Lead sponsor | United Therapeutics |
|---|---|
| Phase | Phase 3 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | treatment |
| Enrollment | 84 |
| Start date | 15 August 2017 |
| Primary completion | 3 December 2019 |
| Estimated completion | 3 December 2019 |
| Sites | 82 locations across United States |
United Therapeutics — full company profile →
Adults 18 to 85, any sex, with Pulmonary Hypertension or Heart Failure With Preserved Ejection Fraction. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
The intent of the 6-Minute Walk Test (6MWT) is to evaluate exercise capacity associated with carrying out activities of daily living.
| Group | Value | 95% CI |
|---|---|---|
| Oral Treprostinil | 21.0 | -195 – 62 |
| Placebo | 23.3 | -140 – 104 |
The NT-proBNP concentration is a biomarker associated with changes in right heart morphology and function.
| Group | Value | 95% CI |
|---|---|---|
| Oral Treprostinil | 9.5 | -133 – 499 |
| Placebo | 11.5 | -231 – 172 |
Clinical worsening was defined as the occurrence of any 1 of the following clinical worsening events: hospitalization due to a cardiopulmonary indication (a non-elective hospitalization lasting at least 24 hours in duration caused by clinical conditions directly related to PH and/or heart failure), outpatient administration of IV diuretics, death (all causes), decrease in 6MWD \>15% from Baseline (or the subject was too ill to walk, and the cause was directly related to the disease under study) at 2 consecutive visits on different days (except Week 24).
| Group | Value | 95% CI |
|---|---|---|
| Oral Treprostinil | 6 | |
| Placebo | 5 | |
| Oral Treprostinil | 35 | |
| Placebo | 38 |
The WHO functional classification ranges from I (subject's disease does not affect daily activities) to IV (subject's disease causes severe impairment).
| Group | Value | 95% CI |
|---|---|---|
| Oral Treprostinil | 1 | |
| Placebo | 1 | |
| Oral Treprostinil | 19 | |
| Placebo | 17 | |
| Oral Treprostinil | 21 | |
| Placebo | 24 | |
| Oral Treprostinil | 0 | |
| Placebo | 1 |
| Group | Value | 95% CI |
|---|---|---|
| Oral Treprostinil | 4 | |
| Placebo | 1 | |
| Oral Treprostinil | 10 | |
| Placebo | 16 | |
| Oral Treprostinil | 15 | |
| Placebo | 11 | |
| Oral Treprostinil | 0 | |
| Placebo | 0 |
Time frame: The Treatment Phase was 24 weeks for each subject. The study was discontinued by the Sponsor on 14 October 2019 due to slow enrollment.. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Oral Treprostinil | Placebo |
|---|---|---|---|
| Fluid overload | Metabolism and nutrition disorders | — | — |
| Cardiac failure acute | Cardiac disorders | — | — |
| Acute kidney injury | Renal and urinary disorders | — | — |
| Atrial fibrillation | Cardiac disorders | — | — |
| Bronchitis viral | Infections and infestations | — | — |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | — | — |
| Fibromyalgia | Musculoskeletal and connective tissue disorders | — | — |
| Gastritis erosive | Gastrointestinal disorders | — | — |
| Hyperkalaemia | Metabolism and nutrition disorders | — | — |
| Hypokalaemia | Metabolism and nutrition disorders | — | — |
| Myalgia | Musculoskeletal and connective tissue disorders | — | — |
| Non-cardiac chest pain | General disorders | — | — |
| Sepsis | Infections and infestations | — | — |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | — | — |
| Cardio-respiratory arrest | Cardiac disorders | — | — |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | — | — |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | — | — |
| Pneumonia | Infections and infestations | — | — |
| Syncope | Nervous system disorders | — | — |
| Reaction | System | Oral Treprostinil | Placebo |
|---|---|---|---|
| Headache | Nervous system disorders | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Fatigue | General disorders | — | — |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | — | — |
| Dizziness | Nervous system disorders | — | — |
| Pain in extremity | Musculoskeletal and connective tissue disorders | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — |
| Oedema peripheral | General disorders | — | — |
| Arthralgia | Musculoskeletal and connective tissue disorders | — | — |
| Flushing | Vascular disorders | — | — |
| Muscle spasms | Musculoskeletal and connective tissue disorders | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | — | — |
| Hypotension | Vascular disorders | — | — |
| Chest pain | General disorders | — | — |
| Abdominal distension | Gastrointestinal disorders | — | — |
| N-terminal prohormone brain natriuretic peptide increased | Investigations | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — |
| Bronchitis | Infections and infestations | — | — |
| Dyspepsia | Gastrointestinal disorders | — | — |
| Pain | General disorders | — | — |
| Weight increased | Investigations | — | — |
| Flatulence | Gastrointestinal disorders | — | — |
| Constipation | Gastrointestinal disorders | — | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — | — |
| Hypokalaemia | Metabolism and nutrition disorders | — | — |
| Pain in jaw | Musculoskeletal and connective tissue disorders | — | — |
| Rash | Skin and subcutaneous tissue disorders | — | — |
| Decreased appetite | Musculoskeletal and connective tissue disorders | — | — |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | — | — |
| Myalgia | Musculoskeletal and connective tissue disorders | — | — |
| Blood creatinine increased | Investigations | — | — |
| Gout | Metabolism and nutrition disorders | — | — |
| Presyncope | Nervous system disorders | — | — |
| Viral upper respiratory tract infection | Infections and infestations | — | — |
Most-reported serious reactions: Fluid overload, Cardiac failure acute, Acute kidney injury, Atrial fibrillation, Bronchitis viral, Dyspnoea, Fibromyalgia, Gastritis erosive.
Data from ClinicalTrials.gov NCT03037580 adverse events section.
This was a multicenter, randomized (1:1; oral treprostinil to placebo), double-blind, placebo-controlled study in subjects with World Health Organization (WHO) Group 2 pulmonary hypertension (PH) associated with heart failure with preserved ejection fraction (HFpEF). Once randomized, subjects took the initial dose of study drug at the study site on the day of randomization. Subjects returned to the study site for visits scheduled at Weeks 6, 12, 18, and 24. The duration of study participation was approximately 28 weeks from Screening until study completion (includes a 30-day Screening Phase and 24-week Treatment Phase). The study was discontinued by the Sponsor on 14 October 2019 due to slow enrollment. As only a small portion of the anticipated total subjects had been enrolled, with many terminating early due to the study termination, there was a limited ability to explore the effect of oral treprostinil in this indication in this study.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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